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Tangier disease

ORPHA:31150DiseaseAutosomal recessiveAdolescent, Adult, Childhood, Infancy, Neonatal

Ассоциированные гены (1)

ABCA1
ATP binding cassette subfamily A member 1
Disease-causing germline mutation(s) in
OMIM: 600046

Фенотипы (22)

Очень частый (80–99%)2
HP:0002155Hypertriglyceridemia
HP:0003146Hypocholesterolemia
Частый (30–79%)12
HP:0000656Ectropion
HP:0000958Dry skin
HP:0001433Hepatosplenomegaly
HP:0002027Abdominal pain
HP:0002460Distal muscle weakness
HP:0002730Chronic noninfectious lymphadenopathy
HP:0003477Peripheral axonal neuropathy
HP:0004943Accelerated atherosclerosis
HP:0005145Coronary artery stenosis
HP:0007133Progressive peripheral neuropathy
HP:0008404Nail dystrophy
HP:0030814Orange discoloured tonsils
Периодический (5–29%)8
HP:0001349Facial diplegia
HP:0001712Left ventricular hypertrophy
HP:0001873Thrombocytopenia
HP:0001903Anemia
HP:0003396Syringomyelia
HP:0007957Corneal opacity
HP:0100546Carotid artery stenosis
HP:0010829Impaired temperature sensition

Эпидемиология (2)

Cases/families
Worldwide
Point prevalence
<1 / 1 000 000
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы